With Regard to Treatment of Inborn Errors of Metabolism (IEM)

question 21

Multiple Choice

With regard to treatment of inborn errors of metabolism (IEM) , which of the following statements, if any, is false?

• A) IEMs are all single gene disorders that have been studied for many decades, leading to the development of successful treatment in all cases.
• B) IEMs can be treated by augmentation therapy, treatment to inhibit positively harmful effects, or by prevention therapy.
• C) Treatment for some individual IEMs can involve both augmentation therapy plus treatment to inhibit positively harmful effects.
• D) Treatment of some IEMs involves artificially forcing an increase in a minor metabolic pathway to counteract a build-up in a toxic metabolite produced by a metabolic block in a major metabolic pathway.

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Definitions:

Prefrontal Cortex

The front part of the frontal lobe of the brain, involved in complex behaviors such as planning, decision making, and moderating social behavior.

Hypothalamus Axons

Axons originating from the hypothalamus, involved in functions like temperature regulation, hunger, and emotional responses.

Postsynaptic Membrane

The membrane of the neuron that receives and responds to neurotransmitters from another neuron at a synapse.

Peptides

Chains of amino acids that are shorter than proteins, which play key roles in biological functions and act as neurotransmitters, hormones, and catalysts.